Oxford Univ spin off gets $20 mn for retinal gene therapy

Oxford University and Wellcome Trust-subsidiary Syncona have formed a firm named Nightstar for developing retinal gene therapies

Good news for eye patients - Oxford University and Wellcome Trust-subsidiary Syncona form Nightstar

Singapore: Syncona, an independent subsidiary of the Wellcome Trust, has made a $19.60 million (£12 million) investment in NightstaRx, which is a spin-out from the University of Oxford and its research commercialization company Isis Innovation. Nightstar will focus on the development and commercialization of therapies for retinal dystrophies, which are degenerative conditions affecting vision.

The company's first programme is a gene therapy for an inherited form of progressive blindness called choroideremia developed by Professor Robert MacLaren at Oxford's Nuffield Laboratory of Ophthalmology. The gene therapy uses a small modified virus, AAV.REP1 to deliver the correct version of the choroideremia (CHM) gene to cells in the retina of the eye.

The vector used to treat choroideremia, AAV.REP1, was developed by a team of researchers, led by Professor Robert MacLaren of the University of Oxford, and is currently being studied in a 12 patient phase I clinical trial supported by the Wellcome Trust and Department of Health.

Mr Chris Hollowood, partner, Syncona, and Nightstar's chairman, commented that, "We are delighted to be working with Professor MacLaren to provide the support required to bring this important therapy to choroideremia patients. We have appointed Dr Melanie Lee as CEO of Nightstar, an experienced industry professional with both scientific and business acumen and we will augment the team over the coming weeks."

Mr Tom Hockaday, MD, Isis Innovation, said that, "The £12 million investment in Nightstar represents one of the largest investments in a new academic spin-out in Europe. Isis Innovation is very excited to have worked with Professor MacLaren since 2009 to protect this technology and we look forward to it benefitting patients."